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1 . Academic Journal
Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access
저자
by
Fumagalli, Francesca
;
Calbi, Valeria
;
Natali Sora, Maria Grazia
;
Sessa, Maria
;
Baldoli, Cristina
, et al.
소스
In
The Lancet
22-28 January 2022 399(10322):372-383
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7 . Academic Journal
Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome.
저자
by
Consiglieri, Giulia
;
Tucci, Francesca
;
De Pellegrin, Maurizio
;
Guerrini, Barbara
;
Cattoni, Alessandro
, et al.
소스
Science Translational Medicine; 5/1/2024, Vol. 16 Issue 745, p1-10, 10p
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7 . Academic Journal
Strategies for single base gene editing in an immortalized human cell line by CRISPR/Cas9 technology.
저자
by
Corrado, Alda
;
Aceto, Romina
;
Miglietta, Simona
;
Silvestri, Roberto
;
Dell'Anno, Irene
, et al.
소스
3 Biotech
. 1/19/2024, Vol. 14 Issue 2, p1-8. 8p.
Web of Science
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7 . Academic Journal
Pro64His (rs4644) Polymorphism Within Galectin-3 Is a Risk Factor of Differentiated Thyroid Carcinoma and Affects the Transcriptome of Thyrocytes Engineered via CRISPR/Cas9 System.
저자
by
Corrado, Alda
;
Aceto, Romina
;
Silvestri, Roberto
;
Dell'Anno, Irene
;
Ricci, Benedetta
, et al.
소스
Thyroid
. Jul2021, Vol. 31 Issue 7, p1056-1066. 11p.
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7 . Academic Journal
Variation rs2235503 C > A Within the Promoter of MSLN Affects Transcriptional Rate of Mesothelin and Plasmatic Levels of the Soluble Mesothelin-Related Peptide.
저자
by
Silvestri, Roberto
;
Pucci, Perla
;
De Santi, Chiara
;
Dell'Anno, Irene
;
Miglietta, Simona
, et al.
소스
Frontiers in Genetics; 8/18/2020, Vol. 11, pN.PAG-N.PAG, 8p
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7 . Academic Journal
Variation rs2235503 C > A Within the Promoter ofMSLNAffects Transcriptional Rate of Mesothelin and Plasmatic Levels of the Soluble Mesothelin-Related Peptide
저자
by
Silvestri, Roberto
;
Pucci, Perla
;
De Santi, Chiara
;
Dell'Anno, Irene
;
Miglietta, Simona
, et al.
소스
FRONTIERS IN GENETICS
; AUG 18 2020, 11 p975 8p.
Scopus
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7 . Academic Journal
Ex vivo hematopoietic stem cell gene therapy for mucopolysaccharidosis type I (Hurler syndrome).
저자
by
Gentner, Bernhard
;
Bernardo, Maria Ester
;
Tucci, Francesca
;
Fumagalli, Francesca
;
Pontesilli, Silvia
, et al.
소스
Molecular Genetics & Metabolism
. Feb2021, Vol. 132 Issue 2, pS42-S43. 2p.
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7 . Academic Journal
Two different strategies of delivery CRISPR/Cas9 system to gene edit rs4644 SNP in LGALS3 gene
저자
by
Corrado, Alda
;
Lepori, Irene
;
Miglietta, Simona
;
Batoni, Simone
;
Vitiello, Marianna
, et al.
소스
CANCER RESEARCH
; JUL 2017, 77 2p. Supplement: 13
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